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A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants With SHANK3 Haploinsufficiency

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Trial Profile

A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants With SHANK3 Haploinsufficiency

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 12 Jul 2025

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At a glance

  • Drugs JAG 201 (Primary)
  • Indications Telomeric 22q13 Monosomy Syndrome
  • Focus Adverse reactions; First in man
  • Sponsors Jaguar Gene Therapy

Most Recent Events

  • 09 Jan 2025 Status changed from not yet recruiting to recruiting.
  • 31 Oct 2024 New trial record

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